EUCTR2018-002433-38-FR进行中（未招募）1 期
A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene - STELLAR
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 18
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •Male or female, = 18 years of age.
- •Clinical presentation consistent with RP with Usher syndrome type 2 or NSRP, based on ophthalmic, audiologic, and vestibular examinations.
- •An ERG result consistent with RP with Usher syndrome type 2 or NSRP.
- •A molecular diagnosis of homozygosity or compound heterozygosity for 1 or more pathogenic exon 13 mutations in the USH2A gene, based on genetic analysis upon Sponsor approval.
- •No limitations to OCT image collection that would prevent high quality, reliable images from being obtained in both eyes (including outer segment [OS] thickness and volume, outer nuclear layer [ONL] thickness, total receptor (TR) thickness, EZ horizontal and vertical widths, apparent continuous EZ area, central macula thickness [CMT], grading of cystic macular lesions [CML] if any), as determined by the reading center.
- •Reliable perimetry measurements in both eyes, as described in the Study Reference Manual and determined by the reading center.
- •Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging, as assessed by the Investigator.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 16
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •Presence of additional non-exon 13 USH2A pathogenic mutation(s) on the USH2A allele carrying the exon 13 mutation in subjects who are compound heterozygous for mutations in exon 13.
- •Presence of non-exon 13 USH2A pathogenic mutation(s) on both USH2A alleles in subjects who are homozygous for mutations in exon 13.
- •Presence of pathogenic mutations in genes (other than the USH2A gene) associated with Usher syndrome Type 2 or NSRP, or other inherited retinal degenerative diseases or syndromes.
- •Any contraindication to IVT injection according to the Investigator’s clinical judgment and international guidelines.
- •Nystagmus or unstable fixation.
- •Amblyopia.
- •Prior receipt of intraocular surgery or procedure or IVT injection within 12 weeks prior to study start or planned intraocular surgery or procedure during the course of the study.
- •Any prior treatment with genetic therapy.
研究者
相似试验
已完成
不适用
A first-in-human study to evaluate the safety and performance of placebo excipient-coated high-density microarray patches applied by an integrated application device.VaccinationsPublic Health - Other public healthACTRN12620000179932Vaxxas Pty Ltd43
已完成
2 期
A first-in-human study to evaluate safety and tolerability of repeated topical administrations of BPR277 ointment in healthy volunteers, and safety, tolerability, and preliminary efficacy of multiple topical administrations of BPR277 in patients with atopic dermatitis and Netherton syndrome (CBPR277X2101)Netherton syndrome10040790NL-OMON40034ovartis Pharma BV5
撤回
不适用
A First-in-Human Study to Assess Safety and Performance of the Cardiac Implants Percutaneous Ring Annuloplasty System in the Treatment of Patients with Functional Tricuspid Regurgitatiofunctional tricuspid regurgitation (TR)10046973NL-OMON55204Cardiac Implants LLC.10
进行中（未招募）
不适用
CBPR277X2101Part 2: Atopic Dermatitis (AD)Part 3: Netherton Syndrome (NS)MedDRA version: 14.1Level: LLTClassification code 10003639Term: Atopic dermatitisSystem Organ Class: 100000004858MedDRA version: 14.1Level: PTClassification code 10062909Term: Netherton's syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-000917-38-DEovartis Pharma Services AG85
进行中（未招募）
不适用
CBPR277X2101EUCTR2011-000917-38-NLovartis Pharma Services AG85